Melody P Chung1, Antonia Valenzuela2, Shufeng Li3, Benjamin Catanese4, Kate Stevens5, David Fiorentino3, Vibeke Strand6, Lorinda Chung6,7. 1. Division of Rheumatology, Johns Hopkins University, Baltimore, MD, USA. 2. Division of Clinical Immunology and Rheumatology, Pontificia Universidad Católica de Chile, Santiago, Chile. 3. Department of Dermatology. 4. Department of Internal Medicine. 5. Department of Radiology, Stanford University School of Medicine, Stanford. 6. Division of Immunology and Rheumatology, Stanford University School of Medicine, Palo Alto. 7. Division of Immunology and Rheumatology, VA Palo Alto Health Care System, Palo Alto, CA, USA.
Abstract
OBJECTIVES: We evaluated the safety and efficacy of oral treprostinil in preventing progression of SSc-associated calcinosis. METHODS: This prospective open-label study enrolled 12 SSc patients meeting 2013 ACR/EULAR classification criteria with confirmed clinical and radiographic evidence of one or more calcinosis deposit in the hands. Patients received oral treprostinil for 1 year. Primary endpoints were safety/tolerability and percentage of patients without radiographic progression of calcinosis at 1 year (<25% increase in Scleroderma Clinical Trials Consortium radiographic score). Secondary endpoints included 1-year changes in Scleroderma HAQ (SHAQ), Cochin Hand Functional Scale, Medical Outcomes Survey Short Form 36 (SF-36), Raynaud Condition Score and patient/physician assessment of calcinosis severity. RESULTS: Twelve female patients were enrolled, half with diffuse cutaneous disease; median age was 55 years (range 35-68 years). Five patients completed the study. Seven patients withdrew due to intolerable adverse effects (n = 3), intercurrent unrelated illness (n = 2, cirrhosis, cancer), progressive SSc (n = 1) and personal reasons (n = 1). Most patients developed headaches and gastrointestinal adverse effects. Four of 11 (36%) patients with 1-year follow-up hand radiographs experienced progression of calcinosis. Of five who completed treatment, calcinosis was stable in four (80%) with progression in one. Based on SF-36 Physical and Mental Component and Domain scores, transition question and SF-6D utility score, all patients who finished the trial reported overall improvement or no change compared with baseline. CONCLUSION: Oral treprostinil was poorly tolerated in SSc patients with calcinosis. Of five patients who completed treatment, most (80%) had documented stability of calcinosis on hand radiographs at 1 year. CLINICALTRIALS.GOV IDENTIFIER: NCT02663895. Published by Oxford University Press on behalf of the British Society for Rheumatology 2021. This work is written by US Government employees and is in the public domain in the US.
OBJECTIVES: We evaluated the safety and efficacy of oral treprostinil in preventing progression of SSc-associated calcinosis. METHODS: This prospective open-label study enrolled 12 SSc patients meeting 2013 ACR/EULAR classification criteria with confirmed clinical and radiographic evidence of one or more calcinosis deposit in the hands. Patients received oral treprostinil for 1 year. Primary endpoints were safety/tolerability and percentage of patients without radiographic progression of calcinosis at 1 year (<25% increase in Scleroderma Clinical Trials Consortium radiographic score). Secondary endpoints included 1-year changes in Scleroderma HAQ (SHAQ), Cochin Hand Functional Scale, Medical Outcomes Survey Short Form 36 (SF-36), Raynaud Condition Score and patient/physician assessment of calcinosis severity. RESULTS: Twelve female patients were enrolled, half with diffuse cutaneous disease; median age was 55 years (range 35-68 years). Five patients completed the study. Seven patients withdrew due to intolerable adverse effects (n = 3), intercurrent unrelated illness (n = 2, cirrhosis, cancer), progressive SSc (n = 1) and personal reasons (n = 1). Most patients developed headaches and gastrointestinal adverse effects. Four of 11 (36%) patients with 1-year follow-up hand radiographs experienced progression of calcinosis. Of five who completed treatment, calcinosis was stable in four (80%) with progression in one. Based on SF-36 Physical and Mental Component and Domain scores, transition question and SF-6D utility score, all patients who finished the trial reported overall improvement or no change compared with baseline. CONCLUSION: Oral treprostinil was poorly tolerated in SSc patients with calcinosis. Of five patients who completed treatment, most (80%) had documented stability of calcinosis on hand radiographs at 1 year. CLINICALTRIALS.GOV IDENTIFIER: NCT02663895. Published by Oxford University Press on behalf of the British Society for Rheumatology 2021. This work is written by US Government employees and is in the public domain in the US.
Authors: Ami A Shah; Elena Schiopu; Soumya Chatterjee; Mary Ellen Csuka; Tracy Frech; Avram Goldberg; Robert Spiera; Stanford L Peng; Ryan J McBride; Jody M Cleveland; Virginia Steen Journal: J Rheumatol Date: 2016-06-15 Impact factor: 4.666
Authors: Emma M Johnstone; Charles E Hutchinson; Andy Vail; Aurelie Chevance; Ariane L Herrick Journal: Rheumatology (Oxford) Date: 2012-08-25 Impact factor: 7.580
Authors: Zhi-Cheng Jing; Keyur Parikh; Tomas Pulido; Carlos Jerjes-Sanchez; R James White; Roblee Allen; Adam Torbicki; Kai-Feng Xu; David Yehle; Kevin Laliberte; Carl Arneson; Lewis J Rubin Journal: Circulation Date: 2013-01-10 Impact factor: 29.690